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Study of Haplo-HSCT + Rivogenlecleucel vs Haplo-HSCT + Post Transplant Cyclophosphamide in Patients With AML or MDS (THRIVE)

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ClinicalTrials.gov Identifier: NCT03699475
Recruitment Status : Terminated (Funding, portfolio re-prioritization)
First Posted : October 9, 2018
Last Update Posted : November 16, 2020
Information provided by (Responsible Party):
Bellicum Pharmaceuticals

Brief Summary:
This study compares the safety and effectiveness of giving rivogenlecleucel (BPX-501 T cells) to patients with AML or MDS post haploidentical hematopoietic stem cell transplant compared to post-transplant cyclophosphamide.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Myelodysplastic Syndromes Biological: rivogenlecleucel Drug: rimiducid Drug: Cyclophosphamide Procedure: haplo-HSCT Phase 2 Phase 3

Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Detailed Description:

In the Phase 2 portion, participants will undergo αβ T cell and CD19+ B cell depleted haploidentical HSCT followed by an infusion of a fixed dose of rivogenlecleucel (BPX-501 T cells) per kg. These participants will be evaluated for prespecified dose limiting toxicities (DLTs) for a 100-day dose limiting toxicity window.

Following completion of the Phase 2 portion, participants will be enrolled and randomized to one of two treatment arms in the Phase 3 portion.

  • Arm A:αβ T-cell and CD19+ B-cell-depleted haplo-HSCT plus treatment with rivogenlecleucel
  • Arm B: haplo-HSCT plus post transplant cyclophosphamide

Pediatric patients ages 12-17 will also be included in US only.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Randomized Phase II/III Study of αβ T Cell-Depleted, Related, Haploidentical Hematopoietic Stem Cell Transplant (Haplo-HSCT) Plus Rivogenlecleucel vs. Haplo-HSCT Plus Post-Transplant Cyclophosphamide (PTCy) in Patients With AML or MDS
Actual Study Start Date : December 27, 2018
Actual Primary Completion Date : July 23, 2019
Actual Study Completion Date : July 23, 2019

Arm Intervention/treatment
Experimental: A: haplo-HSCT plus rivogenlecleucel

αβ T-cell and CD19+ B-cell-depleted haploidentical stem cell transplantation plus rivogenlecleucel

Rimiducid will be administered to inactivate rivogenlecleucel in the event of GVHD not responsive to standard of care treatment

Biological: rivogenlecleucel
Biological: T cells transduced with caspase 9 safety switch
Other Name: BPX-501 T cells

Drug: rimiducid
administered to inactivate rivogenlecleucel in the event of GVHD
Other Name: AP1903

Procedure: haplo-HSCT
treatment for disease

Active Comparator: B: haplo-HSCT followed by cyclophosphamide
haploidentical stem cell transplantation followed by cyclophosphamide post-transplant
Drug: Cyclophosphamide
GVHD prophylaxis
Other Name: Cytoxan

Procedure: haplo-HSCT
treatment for disease

Primary Outcome Measures :
  1. Maximum Allowable Dose/Schedule [Phase 2] [ Time Frame: 100 days ]
    Determine the maximum allowable dose/schedule of rivogenlecleucel and the recommended Phase 3 dose of rivogenlecleucel

  2. Rimiducid Activity [Phase 2] [ Time Frame: 24 months ]
    Assess the activity of Rimiducid in subjects who develop GVHD post rivogenlecleucel administration

  3. Overall survival [Phase 3] [ Time Frame: 3 years ]
    Time from randomization to death due to any cause

Secondary Outcome Measures :
  1. Relapse free survival (RFS) [Phase 3] [ Time Frame: 3 years ]
    Time from randomization to relapse or death from any cause

  2. Graft-versus host disease and relapse-free survival (GRFS) [Phase 3] [ Time Frame: 3 years ]
    Time from randomization until Grade 3-4 acute GVHD; chronic GVHD requiring systemic immunosuppression; disease relapse or death, whichever comes first

  3. Non-relapse mortality (NRM) [Phase 3] [ Time Frame: 3 years ]
    Time from randomization to death without relapse/disease progression

  4. Time to resolution of GVHD after administration of rimiducid [Phase 3] [ Time Frame: 3 years ]
    Resolution of GVHD after administration of rimiducid is defined as complete response or improvement of at least one grade of acute GVHD in patients receiving 1-3 doses of rimiducid

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Signed informed consent

Meeting institutional criteria to undergo allogenic HSCT

Age 18-70 y/o (12-70 y/o in US only)

Patients with AML or MDS as defined below:

AML Patients Patients with intermediate to adverse AML as defined by ELN (Dohner, 2017).

  • AML in first complete remission (CR1) with high-risk features defined as > 1 cycle of induction therapy required to achieve remission OR preceding MDS or myeloproliferative disease
  • AML in CR1 with intermediate-risk features
  • AML in second or subsequent complete response
  • AML with myelodysplasia-related changes (AML-MRC)
  • Therapy related AML in first or subsequent complete remission
  • De novo AML in second or subsequent complete remission

MDS Patients

  • High or very-high risk MDS by IPSS-R classification
  • Intermediate risk or higher MDS patients who failed a hypomethylating agent

Lack of suitable conventional donor (i.e. HLA 10/10 related or unrelated donor)

At least a 5/10 genotypic identical haplotype match

The donor and recipient must be identical, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1, and HLA-DQB1

Patients with adequate organ function

Eastern Cooperative Oncology Group (ECOG) performance status: 0-2

Exclusion Criteria:

  • HLA 10/10 allele matched (HLA-A,-B,-C,-DRBl, and DQB1) related donor or unrelated donor
  • Autologous hematopoietic stem cell transplant ≤ 3 months before enrollment
  • Prior allogeneic transplantation
  • Active CNS involvement by malignant cells (less than 2 months from the conditioning)
  • Current uncontrolled clinically active bacterial, viral or fungal infection
  • Positive HIV serology or viral RNA
  • Pregnancy (positive serum or urine βHCG test) or breast-feeding
  • Fertile men or women unwilling to use effective forms of birth control or abstinence for a year after transplantation
  • Radiographic, histologic, or known history of cirrhosis
  • Overlapping MDS and myeloproliferative neoplasms (MPN) disease
  • Patients with acute promyelocytic leukemia (APL)
  • Known hypersensitivity to dimethyl sulfoxide (DMSO)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03699475

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United States, Tennessee
TriStar Bone Marrow Transplant, LLC
Nashville, Tennessee, United States, 37203
United States, Texas
Methodist Healthcare System of San Antonio Clinical Trials Office
San Antonio, Texas, United States, 78229
Sponsors and Collaborators
Bellicum Pharmaceuticals
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Study Director: Bellicum Pharmaceuticals Bellicum Pharmaceuticals, Inc.
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Responsible Party: Bellicum Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03699475    
Other Study ID Numbers: BPX501-301A
First Posted: October 9, 2018    Key Record Dates
Last Update Posted: November 16, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists